EP4247946A4 - Ribozyme-activated RNA constructs and uses thereof - Google Patents

Ribozyme-activated RNA constructs and uses thereof

Info

Publication number
EP4247946A4
EP4247946A4 EP22737300.8A EP22737300A EP4247946A4 EP 4247946 A4 EP4247946 A4 EP 4247946A4 EP 22737300 A EP22737300 A EP 22737300A EP 4247946 A4 EP4247946 A4 EP 4247946A4
Authority
EP
European Patent Office
Prior art keywords
ribozyme
rna constructs
activated rna
activated
constructs
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
EP22737300.8A
Other languages
German (de)
English (en)
French (fr)
Other versions
EP4247946A2 (en
Inventor
Prashant MALI
Andrew PORTELL
Amir DAILAMY
Aditya Kumar
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of California
University of California Berkeley
University of California, San Diego
Original Assignee
University of California
University of California Berkeley
University of California, San Diego
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of California, University of California Berkeley, University of California, San Diego filed Critical University of California
Publication of EP4247946A2 publication Critical patent/EP4247946A2/en
Publication of EP4247946A4 publication Critical patent/EP4247946A4/en
Withdrawn legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A61K39/215—Coronaviridae, e.g. avian infectious bronchitis virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A61K39/155—Paramyxoviridae, e.g. parainfluenza virus
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/93—Ligases (6)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y605/00—Ligases forming phosphoric ester bonds (6.5)
    • C12Y605/01—Ligases forming phosphoric ester bonds (6.5) forming phosphoric ester bonds (6.5.1)
    • C12Y605/01003—RNA ligase (ATP) (6.5.1.3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53—DNA (RNA) vaccination
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/60—Medicinal preparations containing antigens or antibodies characteristics by the carrier linked to the antigen
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/128—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes processing or releasing ribozyme
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/20011—Coronaviridae
    • C12N2770/20034—Use of virus or viral component as vaccine, e.g. live-attenuated or inactivated virus, VLP, viral protein
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/36011—Togaviridae
    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36141—Use of virus, viral particle or viral elements as a vector
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    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Enzymes And Modification Thereof (AREA)
EP22737300.8A 2021-01-11 2022-01-11 Ribozyme-activated RNA constructs and uses thereof Withdrawn EP4247946A4 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163136201P 2021-01-11 2021-01-11
PCT/US2022/012004 WO2022150773A2 (en) 2021-01-11 2022-01-11 Ribozyme-activated rna constructs and uses thereof

Publications (2)

Publication Number Publication Date
EP4247946A2 EP4247946A2 (en) 2023-09-27
EP4247946A4 true EP4247946A4 (en) 2025-03-26

Family

ID=82358774

Family Applications (1)

Application Number Title Priority Date Filing Date
EP22737300.8A Withdrawn EP4247946A4 (en) 2021-01-11 2022-01-11 Ribozyme-activated RNA constructs and uses thereof

Country Status (4)

Country Link
US (1) US20260115275A1 (enExample)
EP (1) EP4247946A4 (enExample)
JP (1) JP2024502620A (enExample)
WO (1) WO2022150773A2 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115806984B (zh) * 2022-10-18 2023-10-10 昆明理工大学 环状rna及载体和载体的应用
CN116200442A (zh) * 2023-03-17 2023-06-02 清华大学 一种基于毕赤酵母的无细胞合成体系及应用
CN122206801A (zh) * 2023-10-18 2026-06-12 泰思特治疗公司 反式剪接核酸分子及其使用方法

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018237372A1 (en) * 2017-06-23 2018-12-27 Cornell University RNA MOLECULES, METHODS FOR PRODUCING CIRCULAR RNA, AND METHODS OF TREATMENT
WO2021158964A1 (en) * 2020-02-07 2021-08-12 University Of Rochester Ribozyme-mediated rna assembly and expression
WO2022204460A1 (en) * 2021-03-26 2022-09-29 Flagship Pioneering Innovations Vii, Llc Compositions and methods for producing circular polyribonucleotides

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2476707B1 (en) * 2003-11-14 2017-05-24 Children's Medical Center Corporation Self-cleaving ribozymes and uses thereof
JP5913122B2 (ja) * 2010-11-08 2016-04-27 Ｊｃｒファーマ株式会社 新規発現ベクター
MY198399A (en) * 2016-02-02 2023-08-28 Meiragtx Uk Ii Ltd Regulation of gene expression via aptamer-mediated control of self-cleaving ribozymes
SG11202111264TA (en) * 2019-04-12 2021-11-29 Agency Science Tech & Res A ribozyme comprising a target-binding domain
US20220213469A1 (en) * 2019-04-30 2022-07-07 The Broad Institute, Inc. Methods and compositions for barcoding nucleic acid libraries and cell populations
EP3986458A1 (en) * 2019-06-20 2022-04-27 Janssen Sciences Ireland Unlimited Company Self-replicating rna molecules for hepatitis b virus (hbv) vaccines and uses thereof
EP4158017A1 (en) * 2020-05-26 2023-04-05 Shape Therapeutics Inc. Engineered circular polynucleotides
KR20230028375A (ko) * 2020-06-25 2023-02-28 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 원형 rna 번역을 구동하는 유전 요소와 이를 사용하는 방법

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018237372A1 (en) * 2017-06-23 2018-12-27 Cornell University RNA MOLECULES, METHODS FOR PRODUCING CIRCULAR RNA, AND METHODS OF TREATMENT
WO2021158964A1 (en) * 2020-02-07 2021-08-12 University Of Rochester Ribozyme-mediated rna assembly and expression
WO2022204460A1 (en) * 2021-03-26 2022-09-29 Flagship Pioneering Innovations Vii, Llc Compositions and methods for producing circular polyribonucleotides

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
ATSUSHI OGAWA ET AL: "An Artificial Aptazyme-Based Riboswitch and its Cascading System in E. coli", CHEMBIOCHEM, vol. 9, no. 2, 20 December 2007 (2007-12-20), Hoboken, USA, pages 206 - 209, XP055316770, ISSN: 1439-4227, DOI: 10.1002/cbic.200700478 *

Also Published As

Publication number Publication date
WO2022150773A9 (en) 2023-06-01
WO2022150773A2 (en) 2022-07-14
WO2022150773A3 (en) 2022-08-11
US20260115275A1 (en) 2026-04-30
EP4247946A2 (en) 2023-09-27
JP2024502620A (ja) 2024-01-22

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